xenpozymeTM (olipudase Alpha-rpcp) was the first to be approved by the FDA disease specific treatment for ASMD (non-CNS manifestations)
Paris, August 31st2022. The US Food and Drug Administration (FDA) has approved XenpozymeTM (Olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Xenpozyme is the first therapy specifically indicated for the treatment of ASMD and is currently the only approved treatment for this disease.
Bill Sybold
Executive Vice President, Head, Specialty Care at Sanofi
“Sanofi teams have worked to bring hope to patients with ASMD and their families. This is a devastating and extremely rare disease that affects both children and adults xenpozyme represents the culmination of bold work in research and development and our unwavering commitment to this historically overlooked community.”
ASMD, historically known as Niemann-Pick disease types A, A/B and B, is an extremely rare, progressive genetic disorder with significant morbidity and mortality. It has been estimated that fewer than 120 patients have been diagnosed with ASMD in the US. Approximately two-thirds of patients with ASMD in the US are pediatric. Signs and symptoms of ASMD can appear in infancy, childhood, or adulthood and can include an enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other disease manifestations. Traditionally, management of ASMD has included supportive care to address the impact of individual symptoms and careful monitoring to detect potential disease complications.
David Guy
Kaila’s parent, age 16, living with ASMD
“As a young parent, it was devastating at first me and my wife when our daughter Kaila received her ASMD diagnosis. When we first heard the diagnosis, we were faced with so many unknowns: what does it mean, how will it affect them, and most importantly, what hope is…
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